RESEARCH TRIANGLE PARK, N.C., June 06, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that Health Canada has approved oral, once-daily ORLADEYO® (berotralstat) for the routine prevention of recurrent hereditary angioedema (HAE) attacks in patients 12 years and older.
“Today is a momentous day for the Canadian HAE community,” said Jacquie Badiou, president of HAE Canada. “HAE carries with it a severe burden on patients and their families, much of which stems from the unpredictable attacks they experience even when actively managing their disease. We have seen important treatment advancements in recent years, and I believe the first oral preventive therapy for HAE will be a welcome option for many Canadians who are living with this rare condition.”
“I believe this oral, once-daily prophylactic therapy will be a meaningful treatment option for patients who need a prophylactic therapy to help manage their HAE by reducing the frequency of attacks,” said Stephen Betschel, HBSc, MD, FRCPC, FAAAAI, Chair of the Canadian Hereditary Angioedema Network.
“We appreciate Health Canada’s thorough and timely review of ORLADEYO, and with this authorization we are one step closer to bringing this important treatment option to Canadians living with HAE,” said Jared Rhines, general manager of BioCryst Canada. “We look forward to working with stakeholders across the country to ensure these Canadians can receive timely and appropriate access to ORLADEYO. Further, this authorization follows approvals in the US, Europe and other markets, highlighting our ongoing commitment to individuals living with rare diseases regardless of geography.”
ORLADEYO was safe and well tolerated in clinical trials. The most frequently reported adverse reactions in patients receiving ORLADEYO compared with placebo were gastrointestinal reactions. These reactions generally occurred early after initiation of treatment with ORLADEYO, became less frequent with time and typically self-resolved.
About ORLADEYO® (berotralstat)ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.
U.S. Indication and Important Safety Information
INDICATIONORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.
Limitations of useThe safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.
IMPORTANT SAFETY INFORMATION
An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.
The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.
A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C) and in patients taking chronically administered P-glycoprotein (P-gp) or breast cancer resistance protein (BCRP) inhibitors (eg, cyclosporine).
Berotralstat is a substrate of P-gp and BCRP. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.
ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.
The safety and effectiveness of ORLADEYO in pediatric patients